Adeno-associated virus Archives • Fragile X Research

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Fragile X News Today Community • Treatment February 2, 2021April 8, 2022Adeno-associated virus, FDA, mRNAs

The two COVID-19 vaccines that recently received emergency approval from the US and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies.

Gene Therapy Translational Studies for Fragile X Syndrome

FRAXA Research Foundation 2019 Grants • 2020 Grants • 2021 Grants • Beasley, Lindsay • Cincinnati Children's Hospital • Current Research Grants • Erickson, Craig • Genetic Rescue • Gross, Christina • Pedapati, Ernest June 6, 2019March 10, 2021Adeno-associated virus, EEG, NIH

With this $90,000 award from FRAXA Research Foundation, Drs. Ernest Pedapati, Christina Gross, and student Lindsay Beasley will pursue preclinical gene therapy approaches using AAV (adeno-associated virus) vectors for treating Fragile X syndrome at Cincinnati Children’s Hospital. Dr. Craig Erickson elaborates about this in this video.

Role of FMRP in the Regulation of Synaptic Plasticity

FRAXA Research Foundation 1994-2000 Grants • 2001-2005 Grants • 2006-2010 Grants • Completed Research Grants • Greenough, William • University of Illinois at Urbana-Champaign • Weiler, Ivan Jeanne February 2, 2009April 25, 2018Adeno-associated virus, mGluR, mRNAs

With more than $1,000,000 from FRAXA Research Foundation over 13 years, Drs. William Greenough and Ivan-Jeanne Weiler at the University of Illinois uncovered the role of FMRP at synapses, leading to much of the subsequent research on Fragile X syndrome.

FMR1 Gene Delivery Using Herpes Simplex Virus Vectors

FRAXA Research Foundation 2001-2005 Grants • Bloom, David • Completed Research Grants • Genetic Rescue • University of Florida March 1, 2003March 21, 2019Adeno-associated virus

With $89,000 from FRAXA Research Foundation over 2001-2005, Dr. David Bloom investigated gene therapy for Fragile X. The Bloom lab specializes in the development of gene therapy techniques, and they have succeeded in transferring the Fragile X gene (fmr1) into the brains of live mice, using viral vectors. They studied ways to enhance this process, with the ultimate goal of gene therapy for people with Fragile X.

Prospects For Gene Therapy in the Fragile X

FRAXA Research Foundation 1994-2000 Grants • 2001-2005 Grants • Completed Research Grants • Genetic Rescue • New York State Institute for Basic Research • Rattazzi, Mario February 27, 2002August 13, 2018Adeno-associated virus, cancer, TAT (Trans-Activator of Transcription)

With a $90,000 grant from FRAXA Research Foundation from 2000-2002, Dr. Mario Rattazzi at the New York State Institute for Basic Research explored gene therapy: ways to transfer the FMR1 gene across the blood-brain-barrier in normal rats and mice, and then in FMR1 knockout mice. Results published.